Ribozyme in gene therapy research.doc

 PAGE \* MERGEFORMAT 18 Ribozyme in gene therapy research Keywords: ribozyme; gene therapy Abstract: In the early 80s by Cech and Altman American scientists found a ribozyme (Ribozyme), along with in-depth study of human genetic engineering, some of the clinical laboratory workers, and basic science researchers began to notice Ribozym application in gene therapy potential. In recent years, people use Ribozyme can specifically cut the target RNA sequence features, design suitable Ribozyme blocking the expression of specific genes, one after another on AIDS, cancer, reproductive system diseases, hepatitis, leukemia, transplant rejection and other diseases of the large number of studies. Experimental results show that the Ribozyme gene therapy as a way to have a broad application prospect and great clinical value. Advance in Study of Gene Therapy by Ribozyme Ribozyme (Ribozyme) is a kind of endonuclease activity of RNA molecules can specifically cut the target RNA sequence. To date, it was discovered that there are six types of ribozymes, namely, a type intron, RNaseP, hammerhead ribozyme, hairpin ribozyme, HDV ribozyme and the VS ribozyme, but structural point of view the main points into two broad categories [1]: hammerhead ribozyme and hairpin ribozyme. Hammerhead ribozyme is about 30 nucleotides, consists of three base pairs of the helix area, two single-stranded region and bulging of the nucleotide composition. The enzyme can be divided into three parts, the middle is a single-stranded form of the 13 conserved nucleotides, and the composition of the catalytic core of spiral Ⅱ , both sides of the spiral Ⅰ / Ⅲ for variable sequences, together constitute the specific sequence, the decision ribozyme specificity. Hairpin ribozyme cleavage activity required for the minimum length of 50 nucleotides, of which 15 are required. The enzyme from the 4 helix (H) and several ring-areas (J) component. Spiral Ⅰ , Ⅱ ‘s main function is to combine with the target seq